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Miner Moebel Family
My grandson Shepherd has been living with his Duchenne diagnosis for 5 years. Since that day in 2019, our family has turned into crusaders for the Cause. It has been […]
Aviles Family
Our Duchenne journey began like so many others. Gabe, our fourth son, wasn’t reaching his developmental milestones. His doctor told us there was nothing wrong with him, and that, as the […]
Heaton Family
Our son Grant is six years old and was diagnosed with Duchenne Muscular Dystrophy at age three. Duchenne is a genetic disease that destroys muscle, all of them. We are […]
PTC Therapeutics Provides Key Regulatory Updates
PTC Therapeutics Provides Key Regulatory Updates
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy in the United Kingdom
Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for DMD in the UK.
PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients
Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients
Edgewise Therapeutics announced the launch of an educational website dedicated to Becker muscular dystrophy
Edgewise Therapeutics announced the launch of an educational website dedicated to Becker muscular dystrophy